NeuroblastomaPhase 2NCT06057948
A Study of a Vaccine in Combination With Beta-glucan in People With Neuroblastoma
A Phase 2 study testing OPT-821 (QS-21) and oral β-glucan in people with Neuroblastoma and High-risk Neuroblastoma, run by Memorial Sloan Kettering Cancer Center at 7 sites in United States.
Trial summary
The purpose of this study is to test which treatment schedule of β-glucan with bivalent vaccine is more effective for participants with high-risk neuroblastoma that is in complete remission. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Neuroblastoma, High-risk Neuroblastoma, Metastatic Neuroblastoma
- What is being tested?
- OPT-821 (QS-21) and oral β-glucan
- Type of treatment
- Vaccines, Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 7 sitesUnited States
- Study dates
- 21 Sep 2023to 21 Mar 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of NB as defined by international criteria,102 i.e., histopathology (confirmed by the MSK Department of Pathology) or BM metastases plus high urine catecholamine levels, or positivity in MIBG scan.
- HR-NB as defined by risk-related treatment guidelines and international criteria,102 i.e., metastatic/non-localized disease with MYCN amplification (any age), metastatic disease >18 months old, MYCN-amplified localized disease (any age), or disease resistant to standard chemotherapy. For those patients with a positive site of disease that is poorly visualized on a scan, it can be per PI discretion to repeat the scan or proceed with enrollment without a recent scan.
- HR-NB (as defined above) and in 1) first CR at ≥ 6 months from initiation of immunotherapy using anti-GD2 antibody, or 2) second or subsequent CR (achieved after treatment for PD). CR is defined according to the International Neuroblastoma Response Criteria. Patients with positive MIBG scan but negative FDG-PET scan, and CR in BM, are eligible. For those patients with a positive MIBG scan, the negative FDG-PET scan can be per PI discretion to repeat within the 45 day window period prior to enrollment.
- Patients with grade 3 toxicities or less using the Common Toxicity Criteria (Version 5.0) developed by the National Cancer Institute of the USA (CTCAE v5.0) related to hematologic, cardiac, neurological, pulmonary, renal, hepatic or gastrointestinal function as determined by blood tests or physical exam. Plus:
- Absolute neutrophil count (ANC) ≥ 500/mcl
- Absolute lymphocyte count ≥ 500/mcl
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- >21 and <180 days between completion of systemic therapy and 1st vaccination.
- A negative pregnancy test is required for patients with child-bearing capability
- Signed informed consent indicating awareness of the investigational nature of this program.
Who cannot join
- Patients with grade 4 hematologic, cardiac, neurological, pulmonary, renal, hepatic or gastrointestinal function as determined by blood tests or physical exam, using the Common Toxicity Criteria (Version 5.0) developed by the National Cancer Institute of the USA.
- History of allergy to KLH, QS-21, OPT-821, or glucan
- Prior treatment with this vaccine.
- Active life-threatening infection requiring systemic therapy.
- Inability to comply with protocol requirements.
Trial locations and contacts
7 sites in 1 country. Contact the study team or a site near you to ask about taking part.
7 sites in 1 country
United States7 sites, 7 recruiting
- Recruiting
Memorial Sloan Kettering at Basking Ridge (Consent only)
Basking Ridge, New Jersey, 07920
Brian Kushner, MD, 833-675-5491
- Recruiting
Memorial Sloan Kettering Suffolk-Commack (Consent only)
Commack, New York, 11725
Brian Kushner, MD, 833-675-5491
- Recruiting
Memorial Sloan Kettering Westchester (Consent only)
Harrison, New York, 10604
Brian Kushner, MD, 833-675-5491
- Recruiting
Memorial Sloan Kettering Monmouth (Consent Only)
Middletown, New Jersey, 07748
Brian Kushner, MD, 833-675-5491
- Recruiting
Memorial Sloan Kettering Bergen (Consent Only)
Montvale, New Jersey, 07645
Brian Kushner, MD, 833-675-5491
- Recruiting
Memorial Sloan Kettering Cancer Center (All Protocol Activities)
New York, New York, 10065
Brian Kushner, MD, 833-675-5491
- Recruiting
Memorial Sloan Kettering Nassau (Consent Only)
Uniondale, New York, 11553
Brian Kushner, MD, 833-675-5491
About this study
Official title: Phase II Trial of a Bivalent Vaccine With the Immunological Adjuvant OPT-821 (QS-21), in Combination With Randomization of Oral β-glucan, for High-Risk Neuroblastoma
The purpose of this study is to test which treatment schedule of β-glucan with bivalent vaccine is more effective for participants with high-risk neuroblastoma that is in complete remission.
- Being tested
- OPT-821 (QS-21), oral β-glucan
- Keywords
- Neuroblastoma, High-risk Neuroblastoma, Metastatic Neuroblastoma, HR-NB, Memorial Sloan Kettering Cancer Center, 23-198
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OPT-821 (QS-21)Biological
Comprised of the immunological adjuvant OPT-821 (QS-21)
oral β-glucanDietary supplement
Participants will be randomized to receive this agent in two different schedules
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Experimental | Participants will receive oral β-glucan (40 mg/kg/day) for 14 days on, and 14 days off, beginning with vaccination #1 and continuing until vaccination #5 (~20 weeks), then only one 14-day cycle with each of vaccinations #6-#10. Receives: Biological: OPT-821 (QS-21); Dietary Supplement: oral β-glucan |
| Group 2 | Experimental | Participants will receive oral β-glucan (40 mg/kg/day) for 14 days on, and 14 days off, beginning with vaccination #1 and continuing until vaccination #7 (~52 weeks), then only one 14-day cycle with each of vaccinations #8-#10. Receives: Biological: OPT-821 (QS-21); Dietary Supplement: oral β-glucan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean antibody titer in ng/ml of anti-GD2 IgG1 titer
To determine the effect of oral β-glucan schedule on anti-GD2 antibody titers among patients who are in first or second (or later) CR, i.e., have no evidence of neuroblastoma by standard studies.
Measured: up to 32 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2023
- Study start21 Sep 2023
- Primary completion21 Mar 2027 (estimated)
- Study completion21 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- More trials run by Memorial Sloan Kettering Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Brian Kushner, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06057948
- Sponsor's protocol ID
- 23-198
- Status last verified
- Oct 2026
- First submitted
- 21 Sep 2023
- Last updated
- 14 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Trial of a Bivalent Vaccine With the Immunological Adjuvant OPT-821 (QS-21), in Combination With Randomization of Oral β-glucan, for High-Risk Neuroblastoma
- ClinicalTrials.gov ID
- NCT06057948
- Acronym / sponsor ID
- 23-198
- Keywords
- Neuroblastoma, High-risk Neuroblastoma, Metastatic Neuroblastoma, HR-NB, Memorial Sloan Kettering Cancer Center, 23-198
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Study start
- 21 Sep 2023 (actual)
- Primary completion
- 21 Mar 2027 (estimated)
- Study completion
- 21 Mar 2027 (estimated)
- First posted
- 28 Sep 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to test which treatment schedule of β-glucan with bivalent vaccine is more effective for participants with high-risk neuroblastoma that is in complete remission.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1 | Experimental | Biological: OPT-821 (QS-21), Dietary Supplement: oral β-glucanParticipants will receive oral β-glucan (40 mg/kg/day) for 14 days on, and 14 days off, beginning with vaccination #1 and continuing until vaccination #5 (~20 weeks), then only one 14-day cycle with each of vaccinations #6-#10. |
| Group 2 | Experimental | Biological: OPT-821 (QS-21), Dietary Supplement: oral β-glucanParticipants will receive oral β-glucan (40 mg/kg/day) for 14 days on, and 14 days off, beginning with vaccination #1 and continuing until vaccination #7 (~52 weeks), then only one 14-day cycle with each of vaccinations #8-#10. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Diagnosis of NB as defined by international criteria,102 i.e., histopathology (confirmed by the MSK Department of Pathology) or BM metastases plus high urine catecholamine levels, or positivity in MIBG scan.
- HR-NB as defined by risk-related treatment guidelines and international criteria,102 i.e., metastatic/non-localized disease with MYCN amplification (any age), metastatic disease >18 months old, MYCN-amplified localized disease (any age), or disease resistant to standard chemotherapy. For those patients with a positive site of disease that is poorly visualized on a scan, it can be per PI discretion to repeat the scan or proceed with enrollment without a recent scan.
- HR-NB (as defined above) and in 1) first CR at ≥ 6 months from initiation of immunotherapy using anti-GD2 antibody, or 2) second or subsequent CR (achieved after treatment for PD). CR is defined according to the International Neuroblastoma Response Criteria. Patients with positive MIBG scan but negative FDG-PET scan, and CR in BM, are eligible. For those patients with a positive MIBG scan, the negative FDG-PET scan can be per PI discretion to repeat within the 45 day window period prior to enrollment.
- Patients with grade 3 toxicities or less using the Common Toxicity Criteria (Version 5.0) developed by the National Cancer Institute of the USA (CTCAE v5.0) related to hematologic, cardiac, neurological, pulmonary, renal, hepatic or gastrointestinal function as determined by blood tests or physical exam. Plus:
- >21 and <180 days between completion of systemic therapy and 1st vaccination.
Key exclusion criteria (5 listed)
- Patients with grade 4 hematologic, cardiac, neurological, pulmonary, renal, hepatic or gastrointestinal function as determined by blood tests or physical exam, using the Common Toxicity Criteria (Version 5.0) developed by the National Cancer Institute of the USA.
- History of allergy to KLH, QS-21, OPT-821, or glucan
- Prior treatment with this vaccine.
- Active life-threatening infection requiring systemic therapy.
- Inability to comply with protocol requirements.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OPT-821 (QS-21)Comprised of the immunological adjuvant OPT-821 (QS-21) | Biological | Group 1, Group 2 |
| oral β-glucanParticipants will be randomized to receive this agent in two different schedules | Dietary supplement | Group 1, Group 2 |
Primary outcome
Mean antibody titer in ng/ml of anti-GD2 IgG1 titer
Time frame: up to 32 weeks
To determine the effect of oral β-glucan schedule on anti-GD2 antibody titers among patients who are in first or second (or later) CR, i.e., have no evidence of neuroblastoma by standard studies.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2026.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Brian Kushner, MD principal investigator
Memorial Sloan Kettering Cancer Center
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing OPT-821 (QS-21) and oral β-glucan in people with Neuroblastoma and High-risk Neuroblastoma, run by Memorial Sloan Kettering Cancer Center at 7 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Brian Kushner, MD (1-833-MSK-KIDS, kushnerb@MSKCC.ORG), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean antibody titer in ng/ml of anti-GD2 IgG1 titer (time frame: up to 32 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2026.